norcalmom

So I'm thinking who would apply for something like this - and my thought is IVIG manufacturers...like Talecris Biotherapeutics, Inc. - the manufacturers of Gamunex.

Resourses to help all of us.. But the big error was - that RARE disease resources start at less than 4000 in the US per year...not 200 (got that from some other defination of "rare"). so maybe someone in this program can help us... "The Humanitarian Use Device (HUD) program designates a device that is intended to benefit patients by treating or diagnosing a disease or condition that affects fewer than 4,000 individuals in the United States per year as per 21 CFR 814.3(n). " Designating Humanitarian Use Devices (HUDS) The Humanitarian Use Device or HUD program was established in 1990 with passage of the Safe Medical Devices Act and creates an alternative pathway for getting market approval for medical devices that may help people with rare diseases or conditions. As defined by 21 CFR 814.3(n), a HUD is a “medical device intended to benefit patients in the treatment or diagnosis of a disease or condition that affects or is manifested in fewer than 4,000 individuals in the United States per year.” In order to obtain HUD designation, the applicant must provide authoritative references to demonstrate that the device meets the definition of 21 CFR 814.3(n). In addition to the documentation describing the disease or condition, the applicant must also provide the proposed indications for use of the device, and the reasons why such a device is needed for the patient population. One aspect that has becoming increasingly difficult is if the HUD is proposed for an indication that represents a subset of a common disease or condition. In these situations, the applicant must demonstrate that the subset is a medically plausible patient population. A medically plausible subset is considered a regulatory concept where an aspect of the HUD precludes its use in the entire disease or condition. A medically plausible subset is not a readily identifiable subset or a group of patients who meet or do not meet the inclusion and exclusion criteria for a clinical study. Likewise, they are not patients with an unmet medical need. If the HUD application is designated, the applicant can then submit the HDE marketing application to the Center for Devices and Radiological Health (CDRH) for marketing review.

"The ability of a pharmaceutic company to fund research is limited to developing a new drug that may treat an illness " (quote by Allergan exec to Beth Maloney at recent symposium) But - What about the ability to research the imapct of an existing drug on a new audience? I'm assuming that they would be interesting in expanding their sales - so if antibioics or IVIG works for pandas kids - why wouldn't they be researching it? I've been thinking about it for a while. I attended a IDC forum at Stanford for patients with primary immune deficiency or with common variable immune deficiency a couple of weeks ago. I think that there are several resources we've not considered in getting pandas awareness going. We tend to focus on the parents and the primary physicians / specialist - but there are other influencers that may be more effective like: Manufacturers of IVIG, since a hand full of clients means big $ for them, may want to help put some pressure on the insurance providers....anyone have any expertise in this area of work, or connections with the companies that make the drugs...I know they have resources for paitients and working with insurance for that were talked about at that IDF meeting. And What about existing Autoimmune and Rare Disease Resources? The IDF - Immune Def. Foundation the American Autoimmune Related Disease Association http://www.aarda.org/mission_statement.php National Coalition of Autoimmune Patient Groups http://www.aarda.org/issue_display.php?ID=12 Link to phone # for advocay resources http://autoimmunefoundation.org National Organization for Rare Disorders (and yes - pandas is a rare disease if no more than 200 NEW cases per year occur..as far as I know ...that is the case - as much as we "see" it everywhere, until docs DX less than 200 cases last year - then for the time being we may be able to get help from this group and this status helps in getting insurance approval for drugs, and it also helps the pharma companies that have "rare" drug staus drugs get extentions on patents, and other financial incentives from FDA and gov to work on rare stuff (because there's not alot of return on this investment) http://www.rarediseases.org/patients-and-families IVIG is on the ORPHAN Drug list already (for one or two disorders only). If we can get a designation and get on that list that the FDA approves (not the insurance providers) than the insurance providers cannot reject out claims . At least thats how I think it works...more reserach necessary..not that getting FDA to recognize us would be easy, but they do have resources for rare diseases. Getting on the Orphan drug list is a big deal but maybe being added as a disorder treatable by a drug already on the Orphan Drug list would be easier. that's just from 3 minutes of brainstorming. Lets make a list and identify who most likely to have influence and affintiy for pandas. Then we can start contacting them on a more formal basis. If some people want to join in helping me - it would make the process go much faster! We may only find a couple that are a fit, but its worth a few emails and calls. AND - Anyone in the DC area that may be interested in any of this or have some connections?? - there is an upcoming meeting on OCT 12 in DC - I don't think its for us, but the folks there may know more about who can help us. More info on that event here: http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/default.htm

"the ability of a pharmaceutic company to fund research is limited to developing a new drug that may treat an illness " But - What about the ability to research the imapct of an existing drug on a new audience? I'm assuming that they would be interesting in expanding their sales - so if antibioics or IVIG works for pandas kids - why wouldn't they be researching it? So, Alleran may not be the company we need to talk to - but I don't see why resources at the big manufacturers of (esspeically of IVIG, since a hand full of clients means big $ for them) can't put some pressure on the insurance providers....anyone have any expertise in this area of work, or connections with the companies that make the drugs... I'm going to tart another thread on this.

Does your child have lung infection with unclear breathing sounds? There are two ways mycoP can go - just into your lungs (tyrpical walking pneumonia that can be detected early and treated) and chronic - a typical presentation - not in your lungs. If IgG is elevated and does not come down, or continues to rise - you have an active infection - regardless of negative chest Xray or IgM. So - some of it can be detected, but sometimes not. If you have IgM - you have myco P, no need for chest Xray, unless problems with lungs and IgM does not come down with antibiotics (as far as I know). Does your child has these problems? If so, get the X ray...if you doc thinks you need it.

I think I can say without getting anyone in trouble that the majority of presentations were on other autoimmune diseases. From the pandas folks - only new material I saw that could be related to pandas in the shorter term was from Drittan, the host of the seminar, and his info is not specifically related to panadas - but uses his technology to show the BBB of mice after strep infections. He mainly researches stroke, and got drawn into the are of pandas due to the BBB aspect of it (becuase he has ways to actully look at it in live mice). Here is a page on him. In terms of research, he's just starting on pandas. Some of these people work their whole lives on one piece of a puzzle to see it if they can solve it and what it might mean in a bigger picture in terms of health. http://devcell.bio.uci.edu/faculty/dritan-agalliu/ So short tem - nothing new for the majority of the pandas population that I saw. I feel like we were extremely privileged to be there. Even though there were many of the top MS reserachers presented - the invitation was not open to MS patients. The most AMAZING (not that it isn't all amazing) work was presented on a peptide that can block a very specific channel that "over produces bad stuff"(my technical translation) in certain autoimmune disease. About to start human trials in Helsinki Oct 8th. They are starting with MS. But works with a number of other diseases like RA too. Not all, but some. He said he'd test some pandas kids blood for levels of the "bad stuff" and if channel blocking might be appropriate for our group....if a pandas person there gets him some samples - I really hope Dr C was int he room when he said that, a simple communication like that can change the entire game sometimes. The name is George Chandy. Human trials in Helsinki bcs FDA taking way too long - US stuff will happen, but Europe is quicker so they are starting there. 30 years he's worked on this, and the route to where he is should this work in humans the way it does in mice and primates, was serendipitous to say the least. Fortune favors the bold - and the persistent! ITs effect in MS is incredible - because MS paralyzied mice can get up and walk around after taking it bcs the myelin will rebuild itself in many cases.(I think that is mostly true statement...again my translation). here is a press release on that: http://www.sbir.gov/sbirsearch/detail/6533 Cunninghams going to be publishing some papers...no date given, no surprise, didn't share what on other than one study will inculde information from the hundreds of blood samples she took over the years and ran her tests on. Her lab hopes to open early in 2013. Its clear to me that we need to move away from arguing about what causes our kids condition. Like the other autoimmune diseases - first we need to prove it is a disease. Diseases get stuff - like special status with FDA and funding for Rare Diseases research and drug insurance special status etc....so proving it has specific autiommune markers (cunningham's research) and that it has physical proof of attacking a physical organ (agalliu's research, although he's not researching pandas - just strep infected mice at the moment) is really, really important for us. Also - most autoimmune diseases don't know the cause. I had a minor autoimmune thing that happened to me and the docs said - well, in 50% of the cases we know this is relaed to Lupus, Hep C or exposure to amalgam fillings....so they had to test me for Lupus and Hep C to rule them out. But that doesn't mean they didn't treat me!! Because - in 50% of the cases - they had no idea what casues the condition. There is however a test for it, and a very specific autimmune marker for it. But cause? most of the time - they can't track that down. But with a test and a treatment - frankly I didn't care what the cause was (well, unless it was lupus or hep C...) because it didn't matter. And in alot of cases we dont' find strep in our kids, and on top of that - strep is everywhere - so proving the casue is hard hard hard... Hopefully soon we will minimally have a test - and the docs can run that test (cunninghams) use that info to then say - well, in 50% of the cases we thing this is caused by strep - and minimally treat the inciting cause of strep, and maximumly treat the autoimmune sequela. And hopefully, along the way - they will pick up on the other things like mycoplasma and other infections that may be underlying. Just having a test - gives pandas credibility in doctors eyes. Pediatricians can perscribe antibiotics and check for infections that are statistically liked to her tests. It lowers their risk of doing something wrong or without something solid to base decision upon. I left feeling mad at our medical system, but hopeful that the dedicated, persistent, scientists on our side will prevail.

ABSOLUTELY get the lydcocaine (or whatever brand of cream your doc will prescribe) - do BOTH arms - a good thick amount of the stuff - at least 30 minutes prior to the draw. About a 3 " diameter circle. Takes that long to work. Also, you may ask your doc for some kind of med to deal with the stress of that one day (for you!!) (no -seriously - when we went in for IVIG I got pych to give us single doses of clonazapam? - somehting like that - not for me, that was a joke, although probably would have been a good idea since I'm sure DS feeds off my stress of anticipating his behavior). I've even considered asking to get a a does when him when we TELL him he needs to get a draw done. We tell him a day or two before - because the worst thing in his mind is a "surprise" blood draw! Also, try to take him when the lab is not very busy so you aren't in agony in the waiting room. DS no longer has this fear. But it was pretty bad for a couple years. Only got over it when he had a couple in a row that weren't so bad - because of the lydocaine, and using the same person that did the draw the last time - at the same lab. He likes some control over the siutaiona and what to expect. We had a bad one once when we did one arm - and they couldn't get a vein, and had to do the other arm - which was not numb - and also had trouble finding vein. He had a fear prior to that, ...but after that day with - 3 nurses and me in room with him freaking out for 20 minutes - it got pretty bad. He was 12 at the time - almost as big as me, about 5-6 and 125 lbs - He had to have a couple that "weren't so bad" before he started to recover from this fear. And, get every test you even think you may need for the next 3 years!! Good luck.

There is an old thread that references an article on the differnt things that help with gut issues - and saccharomyces boulardi - mentioned above is in it. I now look for this ingredient in probiotics we use, because it is helpful with common gut issues that arise when on antibiotics - like C-diff. I don't know if it is strong enough to treat actual infection, but you could research that.

DS was tested a couple dif time for zinc - first made my radar with a below normal range of 8.6 (9-14.7) This was RBC test. Then I wanted to look further into it -- bcs at the time was wondering if DS could have celiacs (in which case you aren't absorbing enough minerals and may result ins ome being low) and the RBC was 11.3 (9-14.7) and the PLASMA zinc - which is a different measure of absorbtion was 74 ( 25-148) I was told that especially with kids - these levels fluctuate a lot daily. So, getting another test, and getting the plasma test vs the RBC version will give better picture of what situation is.

A recent test we did showed high levels of Kryptopyrrols in DS urine. This condition can cause low amounts of B6 and zinc and other nutrients. I'm just starting to look into it. In doing that I came across some information that ties into the supplements and vitamins that are commonly noted when talking about brain function. (GABA, Sam-e, trytophan, zinc, B-vitamins, copper levels, etc...) I came across a very interesting Dr - Ph.D "doctor" -that researches nutrients and behaviors. Check out the presentation under "Depression" - you have to download the preso.. He also has a bunch of videos (but they aren't really detailed - the presenation is more detailed). I only looked at one preso, but watched most of the videos. He says there are several groups you can break people into, and that various tests will help you figure out what group you are probably in. Strategies for each group are VERY different (sometimes opposite). And many of the diagnostic clues are tests, you may have already done on you kids. Its not perfect, there is some overlap. http://www.walshinstitute.org/MedicalConditions.asp# I've neen thinking about this ever since we started a very low does SSRI - we never ramped it up, just left him at the very, very low does. Didn't see much improvement (if any) - but given that many pandas kids recact badly to it, that has kept me from increasing, as we'll as don't really think this will be a magic bullet for him and may just mask what is going on in his immune system and other stuff we try to figure out what is really going on. At the IOCDF meeting I went to a presentation on neurotransmitters. My take away was that we don't know what most (if any) of these REALLY do. Don't know if they are causal, or a bi-produt of something else. Various symptoms are linked to having too much - and many of those symptoms are linked to having too little. Some are merely "regulators" ..They regulate the other ones...but all the research seemed very very early. DS has had a number of the tests on Dr Walsh's site over the years (like B-12,and zinc), and even the fact that many are in normal range, can help determine what group you belong to and therefore what supplements may work better in your case. My interest in this is twofold - since DS is on long term antibitoics, and I know that it is bad for his gut, and we know the gut is large part of the immune system, I want to do what I can to keep it healthy through out this process. It also beats just trying out various supplements and vitamins randomly to see if they work, or have poor outcome, or no outcome (and even figuring out IF it was the supplement - to determine efficacy of on one kid that has a host of various exposures over a few months time cannot be done with any certainty).